Physicians have long searched for a more effective treatment for Graves’ disease, an autoimmune disorder that leads the thyroid gland to produce more hormones than it should. About half of patients with Graves’ disease also develop thyroid-associated ophthalmopathy, or thyroid eye disease. In that condition, patients frequently experience symptoms such as eye bulging (proptosis), eyelid swelling, constant stare, pain and double vision. They can even go blind.
Traditionally, doctors have turned to oral steroids to reduce inflammation around the eye and artificial tears to relieve dryness. Surgery isn’t usually an option until the disease becomes inactive. Even then, the procedure is costly, complex and not guaranteed to fully restore vision or appearance.
Now a highly anticipated drug could change the treatment paradigm. The drug, teprotumumab, is a monoclonal antibody first developed to treat cancer. It proved an ineffective cancer therapy, but Terry J. Smith, M.D., saw its potential to interrupt the development of Graves’ disease. Dr. Smith’s basic science research laid the groundwork for translating teprotumumab to ocular patient care.
Dr. Smith, who directs the national Graves’ Disease and Thyroid Foundation, is a professor of ophthalmology at Kellogg—the only endocrinologist in the U.S. with a full-time primary faculty appointment at an academic eye center.
Phase 2 and phase 3 clinical trials, directed with co-principal investigator Raymond Douglas, M.D., Ph.D., a Graves’ disease specialist, oculoplastic surgeon, and past Kellogg faculty member, yielded dramatic results. Most trial participants who received intravenous teprotumumab had reduced proptosis, eye pain, double vision and swelling. Many experienced positive results within weeks. The results often lasted for a year or more, with limited, manageable side effects.
After fast-tracking its review, a FDA Advisory Panel recently voted unanimously to recommend approval of teprotumumab with a vote of 12-0 in favor of this breakthrough therapy.
“For the first time, we seem to have a medical therapy that outperforms currently available treatments,” says Dr. Smith. “Teprotumumab may make it possible to treat the disease earlier, reducing inflammation around the eyes and lessening the severity of other symptoms.”